Healthcare Resource Use Associated With Tumor-Induced Osteomalacia: A Literature Review

Abstract

Context: Tumor-induced osteomalacia (TIO) is an ultra-rare, paraneoplastic syndrome caused by tumors that secrete fibroblast growth factor 23 (FGF23). Initial signs and musculoskeletal symptoms can be nonspecific and unrecognized, leading to long delays in diagnosis and treatment, and resulting in severe and progressive disability in patients with TIO.

Objective: This review aimed to identify published evidence on healthcare resource use in TIO to better understand the burden of the disease.

Evidence acquisition: A targeted literature review was conducted to identify publications reporting on disease characteristics and healthcare resource use associated with TIO.

Evidence synthesis: In total, 414 publications were included in the review, of which 376 were case reports. From the case reports, data on 621 patients were extracted. These patients had a mean (SD) age of 46.3 (15.8) years; 57.6% were male. Mean time from first symptoms to diagnosis of TIO was 4.6 (4.7) years and, in cases where imaging tests were reported, patients underwent a mean of 4.1 (2.7) procedures. Tumor resection was attempted in 81.0% of patients and successful in 67.0%. Fracture was reported in 49.3% of patients. Results from association analyses demonstrated that longer time to diagnosis was associated with poorer tumor resection outcomes and a higher probability of tumor recurrence. Unfavorable tumor resection outcomes were associated with greater use of pharmacologic treatment and a greater likelihood of orthopedic surgery.

Conclusion: TIO is associated with a substantial healthcare resource burden. Improvements in the diagnostic process could lead to better management of TIO, thereby benefiting patients and reducing that burden.

Keywords: clinical burden; diagnosis; healthcare resource; resection; tumor-induced osteomalacia.

Figure 1.

PRISMA diagram for the selection of eligible publications identified through MEDLINE.Abbreviations: PRISMA, Preferred Reporting Items for Systematic Reviews and Meta-Analyses; SLR, systematic literature review.

Figure 2.

Visual representation of tumor location by anatomical region and individual site. In the case report studies, tumor locations were reported by site, which are further grouped by anatomical region here.

Figure 3.

Tumor removal and recurrence. Visualization of surgical outcomes and incidence of tumor recurrence from the case report studies data.